Your missing links are here (16 October 2020)

Top Pick

• In the wake of her recent Nobel Prize, Jennifer Doudna is up for more. In a talk with Future Human, she shares her ideas of how much more we can expect from CRISPR in the clinic. Among other visionary ideas, she thinks we will see a move from editing genomes to regulating them instead.

Nobel Prize

• Even though you probably already know everything about CRISPR - the genome editing technology that was the cornerstone of this years' Nobel Prize in Chemistry - you might find it useful or at least entertaining to watch this animated video. It gives an excellent description of what CRISPR is all about and how researchers became aware of its tremendous potential.
• The Nobel Prize is a hugely prestigious award, and many top researchers aspire for it. So it's no wonder that the internet has flourished with speculations about whether others than Emmanuelle Charpentier and Jennifer Doudna deserve a slice of the prize, e.g. Feng Zhang, Virginijus Siksnys, or Rodolphe Barrangou and Philippe Horvath. Read a brief description of who contributed with what in the Wire.

Research

• Researchers at the University of Pittsburgh School of Medicine have developed a porcine model to study the congenital human disease phenylketonuria (PKU). The researchers injected Cas9 and gRNAs into pig zygotes to delete part of the PAH-gene and thus created pigs exhibiting hyperphenylalaninemia.

Delivery & Safety

• Researchers at Kagoshima University in Japan have developed a new AAV2 vector-mediated in vivo delivery system for the treatment of hepatitis B. The system delivers three gRNAs simultaneously and significantly reduced viral episomal DNA in liver cells.

Industry

• ERS Genomics that handle Emmanuelle Charpentier's CRISPR/Cas9 intellectual property has signed a licensing agreement with Japanese biotech company FASMAC that provides genetic analysis reagents and kits and more.

COVID-19

• The genome engineering company Synthego has developed a platform that uses CRISPR to accelerate the study of potential treatment targets for SARS-CoV-2. The platform compares host-virus protein interaction networks and is described in a Science paper that was published yesterday.

Clinical Trials

• The University of Minnesota has opened a Phase II clinical trial for the treatment of metastatic gastrointestinal solid tumour cancer. The trial will use CRISPR to increase the capability of T cells to fight cancer cells by editing the CISH (Cytokine-Induced SH2 protein) intracellular checkpoint.

Reviews

• mRNA CRISPR therapy can be used to replace or edit the CFTR-gene and is a promising treatment for cystic fibrosis. In a recent review in Human Gene Therapy, James Dahlman from Georgia Institute of Technology, USA elaborates on the recent advances.
• British researchers at University of Oxford have reviewed the many different attempts to use CRISPR in the treatment of myotonic dystrophy type 1 (DM1).

Beyond Medicine

• CRISPR has a huge potential in agriculture, but its application is hampered by the difficulties of regenerating plants from cultured cells that are often used for genetic editing. Now researchers have used a GRF–GIF chimeric protein to improve regeneration in both monocots and dicots. This will allow for accelerated crop improvement with CRISPR in the future.